NCIt definition : An orally bioavailable synthetic analog of the neuroactive steroid allopregnanolone
and positive allosteric modulator of the gamma-aminobutyric acid (GABA)-A receptor,
with anxiolytic, sedative, antidepressant and anticonvulsant activities. Upon oral
administration, ganaxolone specifically targets and binds to the allosteric sites
of the synaptic and extrasynaptic GABA-A receptor-chloride ionophore complex in the
central nervous system (CNS). This leads to an increase in the opening of chloride
channels, membrane hyperpolarization, increases the inhibitory effect of GABA on the
CNS, and inhibits neurotransmission. This blocks seizure propagation and elevates
seizure thresholds.;